Cy­to­ki­net­ics gets the Roy­al­ty treat­ment, snag­ging up to $450M to sup­port close­ly watched heart pro­grams

Cy­to­ki­net­ics stum­bled a bit with its close­ly watched lead heart drug over the last year or so, los­ing two phar­ma part­ners af­ter miss­ing a key sec­ondary end­point in a Phase III study. But things are look­ing up in 2022, as Roy­al­ty Phar­ma is reach­ing a lit­tle deep­er in­to its wal­let to bol­ster that pro­gram and an­oth­er heart can­di­date.

Roy­al­ty Phar­ma has agreed to lend up to $300 mil­lion to sup­port the po­ten­tial com­mer­cial­iza­tion of Cy­to­ki­net­ics’ lead can­di­date, ome­cam­tiv mecar­bil, and de­vel­op­ment of its oth­er heart pro­gram, afi­camten. The cash will come in five tranch­es, in­clud­ing an ini­tial tranche of $50 mil­lion up­on clos­ing and four oth­ers up­on cer­tain reg­u­la­to­ry and clin­i­cal mile­stones. Each tranche has an in­ter­est-free and pay­ment-free pe­ri­od of six cal­en­dar quar­ters, fol­lowed by 34 cal­en­dar quar­ters of in­stall­ment re-pay­ments to­tal­ing 1.9 times the amount drawn.

In ad­di­tion, Roy­al­ty is putting down $50 mil­lion up­front and an­oth­er $100 mil­lion in biobucks for a roy­al­ty on afi­camten of 4.5% on sales up to $1 bil­lion and 3.5% on sales above $1 bil­lion, sub­ject to cer­tain po­ten­tial step-downs.

Cy­to­ki­net­ics’ stock $CYTK was down near­ly 4% on Fri­day morn­ing, with shares pric­ing in at $37.25 apiece.

Robert Blum

The deal ex­pands on a “fruit­ful re­la­tion­ship” that dates back to 2017, ac­cord­ing to Cy­to­ki­net­ics CEO Robert Blum, when Roy­al­ty bought a 4.5% roy­al­ty on world­wide sales of ome­cam­tiv mecar­bil for $90 mil­lion and bought an­oth­er $10 mil­lion worth in eq­ui­ty.

“Our his­to­ry has taught us that do­ing a deal opens the door on an­oth­er deal,” Blum said dur­ing an in­vestor call on Fri­day, adding that the com­pa­ny is con­sid­er­ing go­ing af­ter part­ner­ships in oth­er ter­ri­to­ries like Japan and Eu­rope.

Just a cou­ple of weeks ago, the South San Fran­cis­co biotech an­nounced an ex­pand­ed part­ner­ship with Shang­hai biotech Ji Xing Phar­ma­ceu­ti­cals to de­vel­op and com­mer­cial­ize ome­cam­tiv mecar­bil in Chi­na, Hong Kong, Macau and Tai­wan.

“It was our goal to com­plete a struc­tured fi­nanc­ing trans­ac­tion to fur­ther bol­ster our bal­ance sheet to sup­port the de­vel­op­ment and ex­pan­sion of our car­dio­vas­cu­lar pro­grams and po­ten­tial fran­chise,” Blum said. “To­day’s an­nounce­ment puts a punc­tu­a­tion point on a struc­tured fi­nanc­ing deal cam­paign that we launched last year, and which ul­ti­mate­ly had us ac­tive­ly en­gag­ing with over a dozen dif­fer­ent in­vest­ment funds, most of which we took in­to de­tailed dis­cus­sions of terms.”

Fif­teen years af­ter en­ter­ing the clin­ic, Cy­to­ki­net­ics read out the Phase III da­ta for ome­cam­tiv mecar­bil back in No­vem­ber 2020. The drug tech­ni­cal­ly worked, meet­ing its pri­ma­ry end­point, but it flopped on a key sec­ondary end­point — re­duc­tion of car­dio­vas­cu­lar death. Am­gen shrugged off its 14-year al­liance with Cy­to­ki­net­ics just over a week lat­er. Then in May, Astel­las walked out of its own part­ner­ship on skele­tal sar­com­ere ac­ti­va­tors for dis­eases as­so­ci­at­ed with mus­cle weak­ness.

Cy­to­ki­net­ics re­turned that same month with post-hoc da­ta (which can be a dif­fi­cult sell at the FDA) sug­gest­ing ome­cam­tiv mecar­bil works bet­ter in sick­er pa­tients. In the analy­sis, Cy­to­ki­net­ics sep­a­rat­ed pa­tients from the Phase III GALAC­TIC-HF study in­to four quar­tiles based on ejec­tion frac­tion, a mea­sure­ment of how well the left ven­tri­cle pumps blood with each heart­beat. Pa­tients in the low­er two quar­tiles — those with an EF of 22% or low­er, and be­tween 29% to 32% — saw a 15% and 17% rel­a­tive risk re­duc­tion of heart fail­ure events and car­dio­vas­cu­lar death com­bined, Cy­to­ki­net­ics re­port­ed at ACC. No dif­fer­ence was seen in the up­per two quar­tiles.

The com­pa­ny had said it hoped to sub­mit an NDA in Q4 2021, though no fur­ther up­dates have been giv­en.

“We ex­pect to com­ment up­on its po­ten­tial ac­cep­tance with the FDA,” a spokesper­son told End­points News on Fri­day.

Then there’s afi­camten, Cy­to­ki­net­ics’ car­diac myosin in­hibitor tar­get­ing hy­per­trophic car­diomy­opa­thy (HCM). Cy­to­ki­net­ics read out some pos­i­tive Phase II da­ta back in Ju­ly, show­ing pa­tients tak­ing the can­di­date for 10 weeks saw “sub­stan­tial and sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tions” from base­line in the av­er­age rest­ing left ven­tric­u­lar out­flow tract pres­sure gra­di­ent (LVOT-G) and the av­er­age post-Val­sal­va LVOT-G, com­pared to place­bo.

That drug is in main com­pe­ti­tion with Bris­tol My­ers Squibb’s mava­camten, which was hit with a ma­jor set­back in No­vem­ber when the FDA ex­tend­ed its PDU­FA date three months, from Jan. 28 to April 28.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.

J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,700+ biopharma pros reading Endpoints daily — and it's free.